LMNA-related cardiocutaneous progeria syndrome

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Overview

LMNA-related cardiocutaneous progeria syndrome is an extremely rare genetic condition caused by changes (mutations) in the LMNA gene. This gene provides instructions for making proteins called lamins, which form a structural framework inside the nucleus of cells. When this gene is altered, it can lead to problems in multiple body systems, particularly the heart and skin. This syndrome combines features of premature aging (progeria) with heart disease and skin abnormalities. Affected individuals may develop tight, thickened, or aged-looking skin, hair loss or thinning, and changes in body fat distribution. Heart problems are a major concern and can include dilated cardiomyopathy (a weakened, enlarged heart), heart rhythm abnormalities, and heart failure. Other features may include joint stiffness, muscle weakness, and skeletal changes. Because this condition is so rare, there is no specific cure. Treatment focuses on managing symptoms, especially heart-related complications. Regular monitoring by a team of specialists is essential. Medications for heart failure, devices for abnormal heart rhythms, and supportive skin care are part of the management approach. Early diagnosis through genetic testing can help guide treatment and monitoring plans.

Also known as:

Key symptoms:

Aged-looking or tight skinHair loss or thinning hairWeakened and enlarged heart (dilated cardiomyopathy)Abnormal heart rhythmsHeart failureLoss of fat under the skinJoint stiffness or contracturesMuscle weaknessShort stature or growth delaySkeletal abnormalitiesThin or fragile skinPremature aging appearanceNail changes or abnormalities

Clinical phenotype terms (27)— hover any for plain English
Absent eyelashesHP:0000561Coronary artery atherosclerosisHP:0001677Ventricular hypertrophyHP:0001714EmphysemaHP:0002097Alopecia universalisHP:0002289Aortic root aneurysmHP:0002616Mitral valve calcificationHP:0004382Abnormality of the pulmonary arteryHP:0004414Squamous cell carcinoma of the skinHP:0006739Papillary renal cell carcinomaHP:0006766
Inheritance

Autosomal dominant

Passed on from just one parent; each child has about a 50% chance of inheriting it

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for LMNA-related cardiocutaneous progeria syndrome.

View clinical trials →

No actively recruiting trials found for LMNA-related cardiocutaneous progeria syndrome at this time.

New trials open frequently. Follow this disease to get notified.

Search ClinicalTrials.gov ↗Join the LMNA-related cardiocutaneous progeria syndrome community →

No specialists are currently listed for LMNA-related cardiocutaneous progeria syndrome.

View NORD Rare Disease Centers ↗Undiagnosed Disease Network ↗

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Travel Grants

No travel grants are currently matched to LMNA-related cardiocutaneous progeria syndrome.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about LMNA-related cardiocutaneous progeria syndrome

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Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Questions for your doctor

Bring these to your next appointment

  • Q1.What is the current condition of my (or my child's) heart, and how often should it be monitored?,What specific LMNA mutation was found, and does it tell us anything about how the disease might progress?,What medications are recommended, and what side effects should I watch for?,Are there any activity restrictions I should follow because of the heart condition?,Should other family members be tested for this genetic change?,Are there any clinical trials or research studies we could participate in?,What emergency signs should prompt me to go to the hospital immediately?

Common questions about LMNA-related cardiocutaneous progeria syndrome

What is LMNA-related cardiocutaneous progeria syndrome?

LMNA-related cardiocutaneous progeria syndrome is an extremely rare genetic condition caused by changes (mutations) in the LMNA gene. This gene provides instructions for making proteins called lamins, which form a structural framework inside the nucleus of cells. When this gene is altered, it can lead to problems in multiple body systems, particularly the heart and skin. This syndrome combines features of premature aging (progeria) with heart disease and skin abnormalities. Affected individuals may develop tight, thickened, or aged-looking skin, hair loss or thinning, and changes in body fat

How is LMNA-related cardiocutaneous progeria syndrome inherited?

LMNA-related cardiocutaneous progeria syndrome follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.